The clinical effectiveness and cost-effectiveness of primary stroke prevention in children with sickle cell disease: a systematic review and economic evaluation.

Cherry, Mary Gemma; Greenhalgh, J; Osipenko, Leeza; Venkatachalam, M; Boland, Angela; Dündar, Yenal; Marsh, Kevin; Dickson, Rumona; Rees, David C.

doi:10.3310/hta16430

Cited by 34 publications

(28 citation statements)

References 71 publications

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“…Patients with SCD have an approximately 250-times-higher risk of stroke than the general childhood population. 10 Chronic transfusion therapy is the mainstay treatment for primary and secondary neurovascular complications. 2,3,12,22,30,35 Although chronic transfusion therapy prevents initial and recurrent stroke in the majority of patients, some patients on chronic transfusion therapy continue to experience strokes and develop progressive vascular disease.…”

Section: Discussionmentioning

confidence: 99%

Encephaloduroarteriosynangiosis and encephalomyoarteriosynangiosis for treatment of moyamoya syndrome in pediatric patients with sickle cell disease

Griessenauer¹,

Lebensburger

Chua

et al. 2015

PED

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OBJECT Pediatric patients with sickle cell disease (SCD) and moyamoya syndrome (MMS) are at significant risk for cerebrovascular accidents despite chronic transfusion therapy. Encephaloduroarteriosynangiosis (EDAS) and encephalomyoarteriosynangiosis (EMAS) are additional therapeutic options for these patients. To date, the incidence of complications after and efficacy of EDAS and EMAS in stroke prevention in this population have been described in several institutional case series reports, but no randomized prospective trials have been reported. METHODS The authors retrospectively reviewed the cases of all pediatric patients at the University of Alabama at Birmingham with a history of homozygous hemoglobin S (HbS) and sickle cell/β-thalassemia (SB0 thalassemia) and on chronic transfusion therapy, including 14 patients with MMS who underwent EDAS or EMAS. RESULTS Sixty-two patients with SCD and on chronic transfusion therapy were identified. After exclusion of patients on chronic transfusion therapy for indications other than stroke prevention, 48 patients (77.4%) remained. Of those patients, 14 (29.1%) underwent EDAS or EMAS. Nine (18.8%) and 25 (52.1%) patients were on chronic transfusion therapy for primary or secondary stroke prevention, respectively, but did not undergo EDAS or EMAS. The 14 patients with SCD and radiological evidence of MMS and on chronic transfusion therapy for primary or secondary stroke prevention underwent 21 EDAS or EMAS procedures for progressive vascular disease (92.9% of patients), stroke (71.4%), and/or seizure (7.1%). The mean (± SD) time from initiation of chronic transfusion therapy to EDAS or EMAS was 76.8 ± 58.8 months. Complications included 1 perioperative stroke, 1 symptomatic subdural hygroma, 1 postoperative seizure, and 1 case of intraoperative cerebral edema that required subsequent cranioplasty. Before EDAS or EMAS, the stroke rate was calculated to be 1 stroke per 7.8 patient-years. One additional stroke occurred during the follow-up period (mean follow-up time 33.7 ± 19.6 months), resulting in a post-EDAS/EMAS stroke rate of 1 stroke per 39.3 patient-years, a 5-fold reduction compared with that in the pre-EDAS/EMAS period. The patients’ mean pre-EDAS/EMAS HbS level of 29.5% ± 6.4% was comparable to the mean post-EDAS/EMAS HbS level of 25.5% ± 6.1% (p = 0.104). CONCLUSIONS The results of this retrospective case series in a large cohort of pediatric patients with SCD and MMS suggest that EDAS/EMAS provides a stroke-prevention benefit with an acceptably low morbidity rate. Given the combined experience with EDAS and EMAS for this indication at this and other institutions, a prospective clinical trial to assess their efficacy compared with that of chronic transfusion therapy alone is warranted.

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Section: Discussionmentioning

confidence: 99%

Encephaloduroarteriosynangiosis and encephalomyoarteriosynangiosis for treatment of moyamoya syndrome in pediatric patients with sickle cell disease

Griessenauer¹,

Lebensburger

Chua

et al. 2015

PED

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“…They reported incremental cost per stroke averted (CPSA) for blood transfusion plus TCD scans relative to TCD scans alone to be in the range of £87,102 (for age 2–7 years) and £114,142 (for ages 8–18 years). Our CEA resulted in an ICER of US$1,900 (≈£1,214) for HU which represents just 1.4% of the lower range of the CPSA reported by Cherry et al This suggests that HU is very cost‐effective in preventing stroke recurrence.…”

Section: Discussionmentioning

confidence: 53%

“…One study has assessed the cost effectiveness of stroke prevention. Cherry et al used Markov modelling to conduct a CEA of primary stroke prevention in children with SCD identified as high risk based on two criteria: a) Transcranial Doppler (TCD) ultrasonography only and b) TCD combined with blood transfusions . They reported incremental cost per stroke averted (CPSA) for blood transfusion plus TCD scans relative to TCD scans alone to be in the range of £87,102 (for age 2–7 years) and £114,142 (for ages 8–18 years).…”

Section: Discussionmentioning

confidence: 99%

Hydroxyurea use in prevention of stroke recurrence in children with sickle cell disease in a developing country: A cost effectiveness analysis

Cunningham-Myrie

Abdulkadri

Waugh

et al. 2015

Pediatr Blood Cancer

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We undertook a cost effectiveness analysis (CEA) of hydroxyurea (HU) in preventing stroke recurrence and/or death. We followed 43 children with sickle cell disease from 2000 to 2009 after having a first clinical stroke, of whom 10 opted for HU therapy. HU use led to decreased stroke recurrence and death without significantly increasing the annual cost of care per patient (J$83,250 vs. J$76,901, P = 0.491). The incremental cost effectiveness ratio (ICER) for prevention of stroke recurrence amounted to J$169,238 (US$1,900), while that for death prevention equalled J$635,843 (US$7,140). HU may be recommended when safe and affordable transfusion therapy is not feasible.

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“…A disutility factor of 3 -13% was deducted from the total expected utility for patients suffering from stroke as a result of BCVI. [18] Statistical Analysis…”

Section: Costs and Outcomesmentioning

confidence: 99%

Screening for Pediatric Blunt Cerebrovascular Injury: Review of Literature and a Cost-Effectiveness Analysis

Malhotra

Kalra

Goodman

et al. 2015

Journal of Pediatric Surgery

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The clinical effectiveness and cost-effectiveness of primary stroke prevention in children with sickle cell disease: a systematic review and economic evaluation.

Abstract: The National Institute for Health Research Health Technology Assessment programme.

Cited by 34 publications

References 71 publications

Encephaloduroarteriosynangiosis and encephalomyoarteriosynangiosis for treatment of moyamoya syndrome in pediatric patients with sickle cell disease

Encephaloduroarteriosynangiosis and encephalomyoarteriosynangiosis for treatment of moyamoya syndrome in pediatric patients with sickle cell disease

Hydroxyurea use in prevention of stroke recurrence in children with sickle cell disease in a developing country: A cost effectiveness analysis

Screening for Pediatric Blunt Cerebrovascular Injury: Review of Literature and a Cost-Effectiveness Analysis

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